FDA grants “orphan” status to Stamford co.

By Alexander Soule

Published 9:53 am, Wednesday, September 2, 2015

The Food and Drug Administration gave “orphan drug” status to a treatment under development by Loxo Oncology to treat soft tissue sarcoma, a form of cancer in cartilage, fat, muscle and other tissues, with the company’s shares gaining more than 4 percent to $20.89 after the opening bell Wednesday.

Under the orphan-drug designation, the FDA grants exclusive market rights and tax credits to encourage the development of medicine for rare disorders that affect fewer than 200,000 people in the United States.

Stamford-based Loxo (Nasdaq: LOXO) is developing Loxo-101, which it describes as an oral, selective inhibitor of tropomyosin receptor kinase (TRK) signaling molecules that have been implicated in varying forms of cancer. Loxo-101 is currently being evaluated in a phase 1 dose escalation trial for patients with advanced solid tumors, with the company reading phase 2 trials for 2016.

In July, Loxo published in the medical journal Cancer Discovery results of a peer-reviewed clinical response of the first patient enrolled in Loxo’s phase 1 trials, a young woman with advanced soft tissue sarcoma in the lung.

“She experienced substantial tumor regression,” said Jennifer Low, chief medical officer of Loxo, in an August conference call. “Her shortness of breath rapidly resolved and she was able to stop her supplemental oxygen and resume activities of daily living ... (After) four months of treatment her partial response was ongoing, with almost complete tumor disappearance of the largest tumors, and she did not have any adverse events that were attributed to Loxo 101.”